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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States
Susan E Matesanz1, Jonathan B Edelson1, Katherine A Iacobellis1
1Division of Neurology (SEM, JFB); Division of Cardiology (JBE, KAI, EM, CAW-W, KYL), Cardiac Center, the Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine; Leonard Davis Institute Center for Healthcare Economics (JBE); Cardiovascular Outcomes, Quality, and Evaluative Research Center (JBE), University of Pennsylvania, Philadelphia; and Data Science and Biostatistics Unit (OO, HG), Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia.
Insights
Healthcare access for Duchenne and Becker muscular dystrophy (DBMD) patients is limited, with low neurology visit rates. Medicaid insurance significantly reduced the likelihood of consistent follow-up care.
Area of Science:
- Neurology
- Genetics
- Public Health
Background:
- Duchenne and Becker muscular dystrophy (DBMD) are genetic neuromuscular disorders.
- Established standards of care exist, but data on healthcare access are limited.
- This study focuses on outpatient subspecialty care utilization in pediatric DBMD patients.
Purpose of the Study:
- To characterize outpatient subspecialty care utilization in pediatric patients with DBMD.
- To identify factors influencing healthcare access for DBMD patients.
Main Methods:
- Retrospective cohort study using administrative claims data (2013-2018).
- Included male patients aged 1-18 years with DBMD diagnosis codes.
- Analyzed annual neurology visits, other subspecialty follow-up, and corticosteroid coverage.
Main Results:
- 1,386 patients included; disease severity (heart/respiratory failure, technology dependence) increased with age.
- Annual neurology visit rate was 0.36 per person-year and did not vary by age.
- Low corticosteroid use (30% of person-years had PDC ≥20%).
- Medicaid insurance was linked to lower odds of annual neurology follow-up (OR 0.23).
Conclusions:
- Neurology follow-up and corticosteroid use are low in DBMD patients.
- Medicaid status, not age, was associated with reduced neurology care access.
- Barriers to regular follow-up must be identified to improve DBMD patient outcomes.
Background And Objectives:
Standards of care exist to optimize outcomes in Duchenne and Becker muscular dystrophy (DBMD), caused by alterations in the DMD gene; however, there are limited data regarding health care access in these patients. This study aims to characterize outpatient subspecialty care utilization in pediatric patients with DBMD.
Methods:
This retrospective cohort study used administrative claims data from IBM MarketScan Medicaid and Commercial Claims and Encounters Research Databases (2013-2018). Male patients 1-18 years with an ICD-9/10 diagnosis code for hereditary progressive muscular dystrophy between January 1, 2013, and December 31, 2017, were included. Participants were stratified into 3 age cohorts: 1-6 years, 7-12 years, and 13-18 years. The primary outcome was rate of annual neurology visits. Secondary outcomes included annual follow-up rates in other subspecialties and proportion of days covered (PDC) by corticosteroids.
Results:
A total of 1,386 patients met inclusion-347 (25.0%) age 1-6 years, 502 (36.2%) age 7-12 years, and 537 (38.7%) age 13-18 years. Heart failure, respiratory failure, and technology dependence increased with age (p for all<0.05). The rate of neurology visits per person-year was 0.36 and did not differ by age. Corticosteroid use was low; 30% of person-years (1452/4829) had a PDC ≥20%. Medicaid insurance was independently associated with a lower likelihood of annual neurology follow-up (OR 0.23; 95% CI 0.18-0.28).
Discussion:
The rate of annual neurology follow-up and corticosteroid use in patients with DBMD is low. Medicaid insurance status was independently associated with a decreased likelihood of neurology follow-up, while age was not, suggesting that factors other than disease severity influence neurology care access. Identifying barriers to regular follow-up is critical in improving outcomes for patients with DBMD.
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